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Background Our previous studies have shown that muscle‐derived cells, including a population of muscle stem cells, transduced with a retroviral vector expressing bone morphogenetic protein 4 (BMP4) can improve the healing of critical‐size calvarial defects. However, we did not evaluate the...
Background Semliki Forest virus (SFV) vectors have a great potential for the induction of protective immunity in a large number of clinical conditions including cancer. Such a potential accounts for the huge efforts made to improve the in vivo expression from SFV vectors. It is noteworthy that...
We report the design of a unique two‐plasmid production system for the first lentiviral vector to be evaluated in humans, VRX496. VRX496 is an optimized VSV‐G pseudotyped vector derived from HIV‐1 that expresses antisense to the HIV envelope gene. We found that a two‐plasmid approach to...
Background In mouse models of retinopathy of prematurity (ROP) inhibitors of vascular endothelial growth factor (VEGF) functions administered systemically completely block retinal neovascularization. In contrast, selective ocular VEGF depletion has achieved an approx. 50% inhibition of retinal...
Background For many applications, efficient gene therapy will require long‐term, organ‐specific therapeutic gene expression. Lentiviral vectors based on HIV‐1 are promising gene delivery vehicles due to their ability to integrate transgenes into non‐dividing cells. Many experimental...
Background Cell‐based therapies for treating insulin‐dependent diabetes (IDD) can provide a more physiologic regulation of blood glucose levels in a less invasive fashion than insulin injections. Previously, we developed an engineered human enteroendocrine L‐cell model for regulated insulin...
Vectors based on lentiviruses efficiently deliver genes into many different types of primary neurons from a broad range of species including man and the resulting gene expression is long term. These vectors are opening up new approaches for the treatment of neurological diseases such as...
Background Acidic fibroblast growth factor (FGF‐1) has been identified as a potent mitogen for vascular cells, inducing formation of mature blood vessels in vitro and in vivo and represents one of the most promising approaches for the treatment of ischemic cardiovascular diseases by gene...
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